Bayer AG and its wholly owned, independently operated subsidiary BlueRock Therapeutics LP, a clinical stage cell therapy company, announced that its investigational cell therapy, OpCT-001, has received Orphan Drug Designation (ODD) from the US FDA for treating Retinitis Pigmentosa (RP).

OpCT-001 is an investigational induced Pluripotent Stem Cell (iPSC)-derived cell therapy being tested in a phase I/II(a) clinical study (CLARICO) for the treatment of primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that include RP and cone-rod dystrophy. RP is one of the most common types of inherited retinal diseases characterised by the loss of rod and cone cells, known together as photoreceptor cells. OpCT-001 aims to restore vision in people living with RP by replacing lost cells in the retina with new functional cells.

“Receiving ODD for RP is an important milestone for the OpCT-001 programme. We believe that OpCT-001 has great promise as a potential therapeutic option for restoring vision in people living with Retinitis Pigmentosa and other primary photoreceptor diseases and look forward to continuing to work with the FDA on its clinical development,” said Amit Rakhit, MD, MBA, Chief Medical Officer, BlueRock. 

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

“The FDA’s Orphan Drug Designation for OpCT-001 to treat Retinitis Pigmentosa underscores the importance of developing urgently needed innovative therapies for patients living with primary photoreceptor diseases. Together with BlueRock, we are excited to be advancing the first-ever clinical trial for an iPSC-derived cell therapy in this field,” said Christian Rommel, Executive Vice President and Global Head—Research and Development, Pharmaceuticals Division, Bayer.